Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice

A major complication of factor replacement therapy for haemophilia is the development of anti-factor neutralizing antibodies (inhibitors). Here we show that liver gene therapy by lentiviral vectors (LVs) expressing factor IX (FIX) strongly reduces pre-existing anti-FIX antibodies and eradicates FIX...

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Detalles Bibliográficos
Autores principales: Annoni, Andrea, Cantore, Alessio, Della Valle, Patrizia, Goudy, Kevin, Akbarpour, Mahzad, Russo, Fabio, Bartolaccini, Sara, D'Angelo, Armando, Roncarolo, Maria Grazia, Naldini, Luigi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Blackwell Publishing Ltd 2013
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3840485/
https://www.ncbi.nlm.nih.gov/pubmed/24106222
http://dx.doi.org/10.1002/emmm.201302857

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